Positive pre-clinical results for Scholar Rock’s SRK-015 therapy in spinal cord injury

Mouse-model data shows positive results in terms of muscle mass, lipid infiltration and muscle health

Data presented at the 35th Annual National Neurotrauma Symposium (Neurotrauma 2017; 9–12 July, Snowbird, USA) have demonstrated beneficial effects for SRK-015 antibody therapy (Scholar Rock) in a preclinical model of spinal cord injury.

The study results revealed that the company’s proprietary antibody, which can selectively block intramuscular activation of myostatin, improved key characteristics of muscle pathology that resulted from severe contusion injury to the spinal cord.

More specifically, the antibody decreased muscle atrophy, educed fat infiltration into muscle, and improved muscle function. SRK-015 is Scholar Rock’s lead antibody drug candidate which is initially being developed for the improvement of muscle strength and function in patients with spinal muscular atrophy (SMA).

The therapy has been developed in collaboration with The Miami Project to Cure Paralysis, a Center of Excellence at the University of Miami Miller School of Medicine, Miami, USA.

“Maintaining muscle mass both above and below the level of spinal cord lesion is needed to preserve health and function of persons with spinal cord injuries both immediately following injury and throughout the lifespan,” says Mark S Nash, professor, Departments of Neurological Surgery and Physical Medicine & Rehabilitation, lead investigator of the study. “We have been interested in myostatin for some time given its role as key negative regulator of skeletal muscle mass in many diseases and degenerative processes. By selectively inhibiting myostatin activation, SRK-015 offers the opportunity to address secondary health and functional decline associated with the musculoskeletal, neuromuscular and cardioendocrine systems.”

The mouse-model data was presented a poster by Gergory Bigford (Miami, USA):

  • Masses of the soleus and gastrocnemius—below the level of the experimentally-induced spinal cord lesion—in the antibody treated group were significantly greater than the non-treated group following experimental injury, and not significantly different from uninjured control.
  • Lipid infiltration into muscle associated with spinal cord injury was significantly reduced in muscles from the antibody treated group compared to the non-treated group.
  • Improvements in muscle health were associated with improved grip strength and functional scores in antibody-treated animals.


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